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2020

BioMarin Expands Vosoritide Clinical Program

BioMarin announced in November that the Company is expanding its clinical program for vosoritide, an investigational analog of C-type Natriuretic Peptide (CNP), with two new Phase 2 studies. The first study is sponsored by BioMarin to investigate the safety of vosoritide in infants with achondroplasia at risk of life-threatening foramen magnum compression. The second study is an investigator-initiated study sponsored by Children’s National Hospital in Washington, D.C. to investigate vosoritide in children with selected genetic forms of short stature, which together represent addressable patient populations of approximately 275,000.

Food and Drug Administration Accepts BioMarin’s New Drug Application for Vosoritide to Treat Children with Achondroplasia

BioMarin announced in November that the U.S. Food and Drug Administration (FDA) has accepted the New Drug Application (NDA) for vosoritide, an investigational, once daily injection analog of C-type Natriuretic Peptide (CNP) for children with achondroplasia, the most common form of disproportionate short stature in humans. This acceptance by the FDA marks the first marketing application accepted for a treatment for achondroplasia in the United States.

BioMarin Receives FDA Approval of Label Expansion to Allow Maximum Dose of 60 mg for Palynziq® (pegvaliase-pqpz) Injection for Treatment of Adults with PKU

BioMarin announced in October that the U.S. Food and Drug Administration (FDA) has approved the supplemental Biologics License Application (sBLA) to increase the maximum allowable dose of 60 mg with Palynziq® (pegvaliase-pqpz) Injection for treatment of adults with Phenylketonuria (PKU). Previously, the maximum dose was 40 mg. In the Phase 3 PRISM studies, 19% of study participants required a 60 mg dose to achieve adequate response to Palynziq.

Scientific Luminary and MacArthur Fellowship Winner, Kevin Eggan, Ph.D., Joins BioMarin as Head of Research and Early Development

BioMarin announced in October the Company hired scientific luminary and MacArthur Fellowship winner, Kevin Eggan, Ph.D., as Group Vice President, Head of Research and Early Development, effective today. Dr. Eggan brings almost two decades of experience in groundbreaking scientific research in rare neurological disorders.

BioMarin Receives FDA Fast Track Designation for PKU Investigational Gene Therapy, BMN 307

BioMarin, a pioneer in developing treatments for phenylketonuria (PKU) and gene therapies, announced in October that the U.S. Food and Drug Administration (FDA) has granted Fast Track designation to BMN 307, an investigational gene therapy for the treatment of individuals with PKU.

BioMarin Doses First Participant in Global PHEARLESS Phase 1/2 Study of BMN 307 Gene Therapy

BioMarin announced in September that it has dosed the first participant in the global PHEARLESS Phase 1/2 study with BMN 307, an investigational gene therapy for the treatment of individuals with PKU. BMN 307 is an AAV5-phenylalanine hydroxylase (PAH) gene therapy designed to normalize blood phenylalanine (Phe) concentration levels in patients with PKU by inserting a correct copy of the PAH gene into liver cells. BMN 307 will be evaluated to determine safety and whether a single dose of treatment can restore natural Phe metabolism, normalize plasma Phe levels, and enable a normal diet in patients with PKU.

BioMarin Announces Presentation of Vosoritide Phase 3 Data in Children with Achondroplasia at the American Society for Bone and Mineral Research 2020 Annual Meeting

BioMarin announced in September that Lynda Polgreen, MD, MS, Lundquist Institute for Biomedical Innovation at Harbor-UCLA Medical Center, Torrance, CA, USA will present data from the randomized, double-blind, phase 3, placebo-controlled, multicenter trial for vosoritide, an investigational analog of C-type Natriuretic Peptide (CNP), in children aged 5 to 18 years with achondroplasia at the American Society for Bone and Mineral Research (ASBMR) Annual 2020 Meeting. The data will be presented during a virtual oral presentation on Saturday, September 12 at 11:50am ET. Achondroplasia is the most common form of disproportionate short stature in humans.

BioMarin Announces The Lancet Publishes Detailed Vosoritide Phase 3 Data Demonstrating Statistically Significant Increase in Annualized Growth Velocity (AGV) Over 52 Weeks in Children with Achondroplasia

BioMarin announced in September that The Lancet has published online results from a randomized, double-blind, phase 3, placebo-controlled, multicenter trial for vosoritide. The data demonstrated that daily subcutaneous administration of vosoritide to children with achondroplasia resulted in significantly increased growth velocity and height Z scores over baseline after one year of treatment as compared to those who received placebo with similar adverse effect profiles.

BioMarin Submits New Drug Application to U.S. Food and Drug Administration for Vosoritide to Treat Children with Achondroplasia

BioMarin announced in August that it has submitted a New Drug Application (NDA) to the U.S. Food and Drug Administration (FDA) for vosoritide.

BioMarin Receives Complete Response Letter (CRL) from FDA for Valoctocogene Roxaparvovec Gene Therapy for Severe Hemophilia A

BioMarin announced in August that the U.S. Food and Drug Administration (FDA) issued a Complete Response Letter (CRL) to the Company’s Biologics License Application (BLA) for valoctocogene roxaparvovec gene therapy for severe hemophilia A. The FDA issues a CRL to indicate that the review cycle for an application is complete and that the application is not ready for approval in its present form.

European Medicines Agency Validates BioMarin’s Marketing Authorization Application for Vosoritide to Treat Children with Achondroplasia

BioMarin announced in August that the European Medicines Agency (EMA) validated the Company’s Marketing Authorization Application (MAA) for vosoritide.

BioMarin Submits Marketing Authorization Application to European Medicines Agency for Vosoritide to Treat Children with Achondroplasia

BioMarin announced in July that the company submitted a Marketing Authorization Application (MAA) to the European Medicines Agency (EMA) for vosoritide.

BioMarin Promotes Company Veteran, Brian R. Mueller, to Executive Vice President, CFO

BioMarin announced in June the promotions of Brian R. Mueller to Executive Vice President, Chief Financial Officer and of Andrea L. Acosta to Group Vice President, Chief Accounting Officer.

BioMarin Provides Additional Data from Recent 4 Year Update of Ongoing Phase 1/2 Study of Valoctocogene Roxaparvovec Gene Therapy for Severe Hemophilia A in Late-Breaking Oral Presentation at World Federation of Hemophilia Virtual Summit

BioMarin announced in June additional data from its previously reported four-year update of an open-label Phase 1/2 study of valoctocogene roxaparvovec, an investigational gene therapy treatment for severe hemophilia A.

BioMarin Provides Highlights of 4 Years of Clinical Data from Ongoing Phase 1/2 Study of Valoctocogene Roxaparvovec Gene Therapy for Severe Hemophilia A

BioMarin announced in May an update to its previously reported results of an open-label Phase 1/2 study of valoctocogene roxaparvovec, an investigational gene therapy treatment for adults with severe hemophilia A.

BioMarin Extends Gene Therapy Leadership with DiNAQOR in a Preclinical Collaboration and License Agreement to Develop Gene Therapies for Rare Genetic Cardiomyopathies

BioMarin announced in May that the company has entered into a preclinical collaboration and license agreement with DiNAQOR AG (DiNAQOR), a gene therapy platform company, to develop novel gene therapies to treat rare genetic cardiomyopathies.

BioMarin Appoints Global Pharmaceutical Veteran C. Greg Guyer, Ph.D., to Chief Technical Officer, Executive Vice President of Global Manufacturing and Technical Operations, Effective May 4

BioMarin announced in April the appointment of C. Greg Guyer, Ph.D., to Chief Technical Officer, Executive Vice President of Global Manufacturing and Technical Operations.

BioMarin Plans Regulatory Submissions for Marketing Authorization of Vosoritide to Treat Children with Achondroplasia in 3Q 2020 in both US and Europe

BioMarin announced in April that based on recent meetings with health authorities in the US and Europe, the Company plans to submit marketing applications to the US Food and Drug Administration (FDA) and the European Medicines Agency (EMA) in the third quarter of 2020 for vosoritide.

BioMarin’s Biologics License Application for Valoctocogene Roxaparvovec Accepted for Priority Review by FDA with Review Action Date of August 21, 2020

BioMarin announced in February that the U.S. Food and Drug Administration (FDA) has accepted for Priority Review the Biologics License Application (BLA) to the FDA for its investigational AAV5 gene therapy, valoctocogene roxaparvovec, for adults with hemophilia A. This acceptance by the FDA marks the first marketing application accepted for a gene therapy product for any type of hemophilia in the United States.

BioMarin, Pioneer in Phenylketonuria, to Begin Clinical Trial with BMN 307 Gene Therapy

BioMarin announced in January that both the U.S. Food and Drug Administration (FDA) and the Medicines and Healthcare Products Regulatory Agency (MHRA) in the U.K. have granted the Company Investigational New Drug (IND) status and approved its Clinical Trial Application (CTA), respectively, for its investigational gene therapy candidate BMN 307.

BioMarin Announces New England Journal of Medicine Publishes 3 Years of Follow-up Data in Phase 1/2 Study of Valoctocogene Roxaparvovec Gene Therapy for Hemophilia A

BioMarin announced in January that the New England Journal of Medicine (NEJM) published an independently peer-reviewed article on up to three years of data from an ongoing Phase 1/2 study to evaluate safety and efficacy of investigational AAV gene therapy, valoctocogene roxaparvovec, for severe hemophilia A.

2019

BioMarin Announces Ongoing Study Demonstrates Durable Treatment Benefit from Brineura® (cerliponase alfa) for 3 Years

BioMarin announced in February that an ongoing open-label extension study treating patients with Brineura® (cerliponase alfa) continued to show a reduced rate of decline compared to a natural history cohort of CLN2 disease for three years as measured by the CLN2 Clinical Rating Scale.

BioMarin Receives Positive CHMP Opinion in Europe for Palynziq® (pegvaliase Injection) for Treatment of Patients with Phenylketonuria (PKU) Aged 16 and Older

BioMarin announced in March that the Committee for Medicinal Products for Human Use (CHMP), the scientific committee of the European Medicines Agency (EMA), has adopted a positive opinion for the company’s Marketing Authorization Application (MAA) for Palynziq® (pegvaliase) Injection to reduce blood phenylalanine (Phe) concentrations in patients with phenylketonuria (PKU) aged 16 and older, who have inadequate blood Phe control (blood Phe levels greater than 600 micromol/L) despite prior management with available treatment options. In addition, the CHMP noted that the data collected in the Phase 3 trial and extension study was suggestive of an improvement in inattention and mood symptoms.

Forbes Names BioMarin 4th Best Midsize Employer in America

BioMarin announced in April that it has been ranked fourth on Forbes magazine’s 2019 list of “America’s Best Midsize Employers,” increasing from 51st on last year’s 2018 list. BioMarin was ranked first among its peers in the ‘Drugs & Biotechnology’ industry.

European Commission Approves Palynziq® (pegvaliase injection) for Treatment of Phenylketonuria (PKU) in Patients Aged 16 Years or Older

BioMarin announced in May that the European Commission (EC) has granted marketing authorization for Palynziq® (pegvaliase injection) at doses of up to 60 mg once daily, to reduce blood phenylalanine (Phe) concentrations in patients with phenylketonuria (PKU) aged 16 and older, who have inadequate blood Phe control (blood Phe levels greater than 600 micromol/L) despite prior management with available treatment options.

BioMarin Provides 3 Years of Clinical Data from Ongoing Phase 1/2 Study of Valoctocogene Roxaparvovec Gene Therapy for Severe Hemophilia A

BioMarin announced in May an update to its previously reported results of an open-label Phase 1/2 study of valoctocogene roxaparvovec. The three-year update demonstrated that bleed rate control with valoctocogene roxaparvovec 6e13 vg/kg dose was maintained for a third year with a median Annualized Bleed Rate (ABR) of 0 and mean ABR of 0.7 in that year. In addition, Factor VIII levels in the 6e13 vg/kg dose appeared to be approaching a plateau in year three. Factor VIII levels measured with the chromogenic substrate (CS) assay at the end of year three were mean and median of 32.7 IU/dL and 19.9 IU/dl, respectively, compared with mean and median of 36.4 IU/dL and 26.2 IU/dL, respectively, at the end of year two.

BioMarin Announces that Phase 3 Cohort of Valoctocogene Roxaparvovec, Gene Therapy Study in Severe Hemophilia A Met Pre-Specified Criteria for Regulatory Submissions in the U.S. and Europe

BioMarin announced in May that its investigational gene therapy, valoctocogene roxaparvovec, for adults with severe hemophilia A achieved pre-specified clinical criteria for regulatory review in the U.S. and Europe.

BioMarin Announces Approval of Vimizim® (elosulfase alfa) in China for Treatment of Morquio A Syndrome

BioMarin announced in June that Vimizim® (elosulfase alfa) has been approved by the National Medical Products Administration (NMPA) for the treatment of patients with mucopolysaccharidosis type IVA (MPS IVA), also known as Morquio A syndrome. Vimizim is the first treatment in China approved for this condition.

BioMarin Announces New England Journal of Medicine Publishes Vosoritide Phase 2 Study Showing Sustained Annualized Growth Up to 42 Months in Children with Achondroplasia

BioMarin announced in June that the New England Journal of Medicine (NEJM) published online results from a Phase 2 dose-finding and extension study for vosoritide.

BioMarin Earns Milestone Payments from Pfizer for TALZENNA® (talazoparib) for Metastatic Breast Cancer Patients with an Inherited BRCA Mutation

BioMarin Pharmaceutical Inc. (Nasdaq:BMRN) today announced that the Company earned a $15 million milestone payment from Pfizer, Inc. This milestone payment was triggered by the European Commission (EC) approval of TALZENNA® (talazoparib) as monotherapy for the treatment of adult patients with germline breast cancer susceptibility gene (gBRCA) 1/2-mutations, who have human epidermal growth factor receptor 2-negative (HER2-) locally advanced (LA) or metastatic breast cancer (MBC).

BioMarin Plans Regulatory Submissions for Marketing Authorization of Valoctocogene Roxaparvovec to Treat Severe Hemophilia A in 4Q 2019 in both U.S. and Europe

BioMarin announced in July that based on recent meetings with health authorities in the U.S. and Europe, the company plans to submit marketing applications to both the U.S. Food and Drug Administration (FDA) and the European Medicines Agency (EMA) in 4Q 2019 for its investigational gene therapy, valoctocogene roxaparvovec, for adults with severe hemophilia A.

BioMarin Appoints Pharmaceutical Veteran and Former J & J Executive, Liz McKee Anderson, to Board of Directors

BioMarin announced in July the appointment of pharmaceutical veteran and former Johnson & Johnson executive, Liz McKee Anderson, to its Board of Directors effective July 15th, 2019. Ms. Anderson serves on the boards of a number of life sciences companies. Before retiring, Ms. Anderson spent 11 years in executive roles of increasing responsibility at Johnson & Johnson.

BioMarin, Pioneer in Phenylketonuria (PKU) Therapies, Submits Clinical Trial Application (CTA) in U.K. for Investigational Gene Therapy for PKU

BioMarin announced in September that it had submitted a Clinical Trial Application (CTA) with the Medicines and Healthcare Products Regulatory Agency (MHRA) in the U.K. for BMN 307, an investigational AAV5-phenylalanine hydroxylase (PAH) gene therapy designed to reduce blood phenylalanine (Phe) concentrations levels in patients with PKU.

BioMarin Announces Cumulative Additional Height Gain of 9.0 cm over 54 months versus Natural History in Children with Achondroplasia Treated with Vosoritide in Phase 2 Study

BioMarin provided an update on its clinical program for vosoritide, an analog of C-type Natriuretic Peptide (CNP), in children with achondroplasia, the most common form of disproportionate short stature in humans, at its annual R&D Day.

BioMarin Submits Marketing Authorization Application to European Medicines Agency for Valoctocogene Roxaparvovec to Treat Severe Hemophilia A

BioMarin announced in November that the company submitted a Marketing Authorization Application (MAA) to the European Medicines Agency (EMA) for its investigational gene therapy, valoctocogene roxaparvovec, for adults with severe hemophilia A.

BioMarin Announces Positive Final Results from Placebo-Controlled Phase 3 Data in Children with Achondroplasia Treated with Vosoritide

BioMarin reported in December positive final results from its randomized, double-blind, placebo-controlled Phase 3 study evaluating the efficacy and safety of vosoritide. The placebo-adjusted change from baseline in growth velocity after one year of treatment with vosoritide, the primary endpoint, was 1.6 cm/yr (p<0.0001). The results were consistent across the broad patient population studied. Vosoritide was generally well tolerated with no clinically significant blood pressure decreases.

European Medicines Agency Validates BioMarin’s Marketing Authorization Application for Valoctocogene Roxaparvovec to Treat Severe Hemophilia A

BioMarin announced in December that the European Medicines Agency (EMA) validated the Company’s Marketing Authorization Application (MAA) for its investigational gene therapy, valoctocogene roxaparvovec, for adults with severe hemophilia A.

BioMarin Submits Biologics License Application to U.S. Food and Drug Administration for Valoctocogene Roxaparvovec to Treat Hemophilia A

BioMarin announced in December that the company had submitted a Biologics License Application (BLA) to the U.S. Food and Drug Administration (FDA) for its investigational AAV gene therapy, valoctocogene roxaparvovec, for adults with hemophilia A.

2018

WORLDSymposium Recognizes BioMarin’s Brineura® (cerliponase alfa) with 2018 New Treatment Award

BioMarin announced in February that The WORLDSymposium 2018 awarded Brineura® (cerliponase alfa) the New Treatment Award, which recognizes important achievements in therapeutic advancements for lysosomal diseases. The award was presented on Monday, February 5.

BioMarin Presents Interim Data of Phase 1/2 Study of BMN 250 for Treatment of Sanfilippo B Syndrome (MPS IIIB) at WORLDSymposium™ 2018

BioMarin announced in February that it presented interim data from a Phase 1/2 trial for BMN 250, an investigational enzyme replacement therapy using a novel fusion of recombinant human alpha-N-acetylglucosaminidase (NAGLU) with a peptide derived from insulin-like growth factor 2 (IGF2), for the treatment of Sanfilippo B syndrome or mucopolysaccharidosis IIIB (MPS IIIB) at WORLDSymposium™ 2018.

Rare Disease Day 2018: BioMarin Launches RARE Scholars Scholarship Program

BioMarin announced in February the launch of RARE Scholars, an annual scholarship for students living with rare disease. RARE Scholars aims to empower patients with continued education by recognizing students living with rare diseases who have demonstrated leadership and participation in school and community activities. The RARE Scholars program will distribute up to $20,000 each year in June, awarding $5,000 for undergraduate four-year or graduate studies or $2,500 for two-year or vocational-technical studies.

BioMarin’s Gene Therapy Manufacturing Facility Recognized with Industry Award

BioMarin announced in March that the International Society for Pharmaceutical Engineering (ISPE) selected the company’s gene therapy manufacturing facility as the 2018 Facility of the Year Category Winner for Project Execution. The recognition highlighted the company’s successful construction of the facility in Novato, CA, which took less than a year to transform basic infrastructure into one of the first gene manufacturing facilities of its kind in the world. Winners were announced on Tuesday, March 20 during the 2018 Europe Annual Conference in Rome, Italy.

European Medicines Agency (EMA) Accepts BioMarin’s Marketing Application for Pegvaliase MAA for Treatment of Phenylketonuria (PKU)

BioMarin announced in March that the European Medicines Agency (EMA) has accepted BioMarin’s submission of a Marketing Authorization Application (MAA) for pegvaliase, a PEGylated recombinant phenylalanine ammonia lyase enzyme product, for the treatment of adults with phenylketonuria (PKU) who have inadequate blood phenylalanine control (blood phenylalanine levels greater than 600 micromol/l) despite prior management with available treatment options including sapropterin.

New England Journal of Medicine Published Open-label Study Showing Brineura® (cerliponase alfa) Reduced the Rate of Clinical Decline of Children with CLN2 Disease, a Form of Batten Disease

BioMarin announced in April that the New England Journal of Medicine (NEJM) published updated results from a multi-center, open-label, dose-escalation and ongoing extension study evaluating the efficacy and safety of Brineura® (cerliponase alfa) in children with CLN2 disease in the May 2018 issue. The new data demonstrated that treatment with Brineura resulted in less decline in motor and language function compared to historical controls.

BioMarin Named To Forbes List Of America’s Best Mid-size Employers

BioMarin announced in May that it had been ranked 51st overall among 500 companies on Forbes magazine’s 2018 list of “America’s Best Mid-size Employers,” and third among companies in the biotechnology industry.

BioMarin Announces First Patient Dosed in Phase 1/2 Study Evaluating Valoctocogene Roxaparvovec Gene Therapy in Severe Hemophilia A Patients with Pre-existing AAV5 Antibodies

BioMarin announced in May that it had dosed the first patient in a Phase 1/2 study (BMN 270-203) evaluating its investigational gene therapy, valoctocogene roxaparvovec, in severe hemophilia A patients with pre-existing AAV5 antibodies.

BioMarin Provides 2 Years of Clinical Data in 6e13 vg/kg Dose from Ongoing Phase 1/2 Study in Valoctocogene Roxaparvovec Gene Therapy for Severe Hemophilia A at World Federation of Hemophilia 2018 World Congress

BioMarin announced in May an update to its previously reported results of an open-label Phase 1/2 study of valoctocogene roxaparvovec (formerly BMN 270), an investigational gene therapy treatment for severe hemophilia A. The updated results were presented during an oral presentation at the World Federation of Hemophilia (WFH) 2018 World Congress in Glasgow, Scotland by John Pasi, M.B., Ch.B., Ph.D., from Barts and the London School of Medicine and Dentistry and primary investigator for this Phase 1/2 study.

BioMarin Receives Standard Approval for Palynziq™ (pegvaliase-pqpz) Injection for Treatment of Adults with Phenylketonuria (PKU), a Rare Genetic Disease

BioMarin announced in May that it had received standard approval from the U.S. Food and Drug Administration (FDA) for Palynziq™ (pegvaliase-pqpz) Injection to reduce blood phenylalanine (Phe) concentrations in adult patients with phenylketonuria (PKU), who have uncontrolled blood Phe concentrations greater than 600 micromol/L on existing management. Palynziq, a PEGylated recombinant phenylalanine ammonia lyase enzyme, is the first approved enzyme substitution therapy to target the underlying cause of PKU by helping the body to break down Phe. Palynziq is BioMarin’s second approved treatment for this important condition.

BioMarin Receives Milestone Payments from Pfizer for Talazoparib

BioMarin announced in June that the Company received $20 million in milestone payments from Pfizer Inc. These milestone payments were triggered by the U.S. Food and Drug Administration (FDA) acceptance of Pfizer’s New Drug Application (NDA) submission for talazoparib and by the European Medicines Agency (EMA) acceptance of Pfizer’s submission of a Marketing Authorization Application (MAA) for talazoparib. These milestone payments are part of an agreement made with Medivation, Inc. when the company purchased talazoparib. Medivation was acquired by Pfizer.

BioMarin Doses First Participant in Phase 2 Study of Vosoritide for Treatment of Infants and Young Children with Achondroplasia

BioMarin announced in June that the company dosed the first participant in a global Phase 2 study for vosoritide, an analog of C-type Natriuretic Peptide (CNP), in infants and young children with achondroplasia, the most common form of disproportionate short stature in humans.

BioMarin Announces First Recipients of RARE Scholars Scholarship Program

BioMarin announced in June that it has awarded the first five recipients of the RARE Scholars program, an annual scholarship for students living with rare disease. The RARE Scholars program provides up to $20,000 to students living with mucopolysaccharidoses, phenylketonuria or Batten disease.

BioMarin Partners with Believe Limited for ‘Breaking Through!’ Musical Theater Intensive

BioMarin announced an exclusive partnership with Believe Limited to produce the ‘Breaking Through!’ musical theater intensive. The first-of-its-kind program is a three-day music workshop for the bleeding disorders community and is designed to provide young adults with powerful education on the healing and therapeutic power of the arts and self-expression. The musical workshop, directed by hemophilia advocate and Believe Limited CEO Patrick James Lynch, will be held from November 9 – November 12, 2018 and will culminate in a Broadway-style performance for local bleeding disorder community members, family and friends.

Forbes Names BioMarin Among World’s Best Employers

BioMarin announced in October that Forbes’ named the company to its list, Global 2000: World’s Best Employers. It is ranked within the top 300 out of a total of 2000 companies and fifth among the nine biotech companies also named. This marks the second time this year that BioMarin has been acknowledged as a top place to work by Forbes, having also been named to the magazine’s list of Best Mid-Sized Employers, earlier this year with a ranking of 51 among 500 companies and third among biotech companies also named.

BioMarin and Believe Limited Announce Selection of 25 High School Students to Perform in First-of-its-Kind ‘Hemophilia: The Musical’

BioMarin announced in October the twenty-five high school students from across the U.S. who will participate in ‘Hemophilia: The Musical,’ a first-of-its-kind theatrical production written and informed by their unique experiences of life as a young person with a bleeding disorder. The musical will take place at New World Stages on November 12, 2018 at 1 pm ET. A live stream of the performance will be made available at BreakingThroughHemophilia.com.

BioMarin Receives Milestone Payments from Pfizer for Talzenna® (Talazoparib) for Metastatic Breast Cancer Patients with an Inherited BRCA Mutation

BioMarin announced in October that the Company earned $15 million in milestone payments from Pfizer Inc. These milestone payments were triggered by the U.S. Food and Drug Administration (FDA) approval of Talzenna® (talazoparib) for the treatment of adult patients with deleterious or suspected deleterious germline BRCA (gBRCA)-mutated, HER2-negative locally advanced (LA) or metastatic breast cancer (MBC). Patients are selected for therapy based on an FDA-approved companion diagnostic.

BioMarin and Believe Limited Announce the Debut of ‘Hemophilia: The Musical’

BioMarin announced in November the debut of the Broadway-style ‘Hemophilia: The Musical,’ a first-of-its-kind theatrical production featuring 25 students affected by a bleeding disorder. A recording of the six-song performance is now available at BreakingThroughHemophilia.com.

2017

BioMarin Receives Access to Priority Medicines (PRIME) Regulatory Support from EMA for BMN 270 Gene Therapy in Hemophilia A

BioMarin announced in February that the European Medicines Agency (EMA) has granted access to its Priority Medicines (PRIME) regulatory initiative for the company’s investigational gene therapy treatment for severe hemophilia A, BMN 270. To be accepted for PRIME, an investigational therapy has to show its potential to benefit patients with unmet medical needs based on early clinical data.

BioMarin Announces Kuvan® (sapropterin dihydrochloride) Patent Challenge Settlement

BioMarin announced in April that it has entered into a settlement agreement with Par Pharmaceutical that resolves patent litigation in the United States (U.S.) related to BioMarin’s Kuvan® (sapropterin dihydrochloride) 100mg oral tablets and powder for oral solution in 100mg packets.

BioMarin Receives Positive CHMP Opinion in Europe for Brineura™ (cerliponase alfa) for First Treatment of CLN2 Disease, a Form of Batten Disease and Ultra-Rare and Fatal Brain Disorder in Children

BioMarin announced in April that the Committee for Medicinal Products for Human Use (CHMP), the scientific committee of the European Medicines Agency (EMA), has adopted a positive opinion for the company’s Marketing Authorization Application (MAA) for Brineura™ (cerliponase alfa) to treat children with Neuronal Ceroid Lipofuscinosis Type 2 (CLN2) disease, a form of Batten disease, which is also known as tripeptidyl peptidase 1 (TPP1) deficiency.

FDA Approves Brineura™ (cerliponase alfa) for the Treatment of CLN2 Disease, a Form of Batten Disease and Ultra-Rare Pediatric Brain Disorder in Children

BioMarin announced in April that the U.S. Food and Drug Administration (FDA) approved Brineura™ (cerliponase alfa) to slow the loss of ambulation in symptomatic pediatric patients 3 years of age and older with late infantile neuronal ceroid lipofuscinosis type 2 (CLN2), also known as tripeptidyl peptidase 1 (TPP1) deficiency. Brineura is the first treatment approved to treat children with CLN2 disease, a form of Batten disease.

European Commission Approves Brineura™ (cerliponase alfa), the First Treatment for CLN2 Disease, a Form of Batten Disease and Ultra-Rare Brain Disorder in Children

BioMarin announced in June that the European Commission (EC) has granted marketing authorization for Brineura™ (cerliponase alfa), the first treatment approved in the European Union for the treatment of neuronal ceroid lipofuscinosis type 2 (CLN2), also known as tripeptidyl peptidase 1 (TPP1) deficiency. The dosing administration includes all ages from birth.

BioMarin Submits Pegvaliase Biologics License Application (BLA) to the U.S. FDA for Treatment of Phenylketonuria (PKU)

BioMarin announced that the company submitted a Biologics License Application (BLA) on Friday, June 30, 2017 to the U.S. Food and Drug Administration (FDA) for pegvaliase, a PEGylated recombinant phenylalanine ammonia lyase enzyme product, to reduce blood phenylalanine (Phe) levels in adult patients with PKU who have uncontrolled blood Phe levels on existing management.

BioMarin’s Investigational Gene Therapy for Hemophilia A at 6e13 vg/kg Dose Maintains Average Factor VIII Levels within Normal Range for over One Year

BioMarin announced in July an update to its previously reported interim results of an open-label Phase 1/2 study of BMN 270, an investigational gene therapy treatment for severe hemophilia A.

Sarepta Therapeutics and BioMarin Pharmaceutical Inc. Announce Execution of a Global Settlement and a License Agreement Resolving Exon Skipping Patent Litigation

Sarepta Therapeutics and BioMarin announced in July that they had executed a license agreement that provides Sarepta Therapeutics with global exclusive rights to BioMarin’s DMD patent estate for EXONDYS 51 and all future exon-skipping products. BioMarin retains the right to convert the license to a co-exclusive right in the event it decides to proceed with an exon-skipping therapy for DMD. In addition, Sarepta and BioMarin executed a settlement agreement, resolving the ongoing worldwide patent proceedings related to the use of EXONDYS 51 and all future exon-skipping products for the treatment of DMD.

BioMarin Announces Plans to Progress Both the 6e13vg/kg and 4e13 vg/kg Doses of BMN 270, its Investigational Gene Therapy for Hemophilia A, into Phase 3 Studies

BioMarin announced in August that it will expand its development plan for BMN 270, its investigational gene therapy for hemophilia A, to include an additional Phase 3 study of the 4e13 vg/kg dose based on updated data as of July 28, 2017 from its ongoing open-label Phase 1/2 study of BMN 270.

Forbes Ranks BioMarin 12th Most Innovative Company in the World

BioMarin announced in August that it has been ranked 12th on Forbes magazine’s 2017 list of the “World’s Most Innovative Companies.”

FDA Accepts BioMarin’s Pegvaliase Biologics License Application (BLA) and Grants Priority Review Designation

BioMarin announced in August that the U.S. Food and Drug Administration (FDA) has accepted for Priority Review the Biologics License Application (BLA) for pegvaliase, a PEGylated recombinant phenylalanine ammonia lyase enzyme product, to reduce blood phenylalanine (Phe) levels in adult patients with phenylketonuria (PKU) who have uncontrolled blood Phe levels on existing management.

BioMarin Presents Interim Data of Phase 1/2 Study of BMN 250 for Treatment of Sanfilippo B Syndrome (MPS IIIB) at 13th International Congress of Inborn Errors of Metabolism (ICIEM) 2017

BioMarin announced in September that it presented interim data from the dose escalation arm of a Phase 1/2 trial for BMN 250, an investigational enzyme replacement therapy using a novel fusion of recombinant human alpha-N-acetylglucosaminidase (NAGLU) with a peptide derived from insulin-like growth factor 2 (IGF2), for the treatment of Sanfilippo B syndrome or mucopolysaccharidosis IIIB (MPS IIIB) at the 13th International Congress of Inborn Errors of Metabolism (ICIEM) 2017.

BioMarin Appoints Robert J. Hombach, Former Baxalta CFO and COO, to Board of Directors

BioMarin announced in October the appointment of former Baxalta Chief Financial Officer and Chief Operations Officer, Robert J. Hombach, to its Board of Directors.

Popular Science Names BioMarin’s Brineura® (cerliponase alfa) One of the Top Health Innovations of 2017 with “Best of What’s New” Award

BioMarin announced in October that Brineura® (cerliponase alfa) has been awarded the 2017 Popular Science “Best of What’s New” award in the health category. Each year, Popular Science reviews thousands of new products and innovations across 11 categories for its annual “Best of What’s New” issue, selecting those that represent a significant step forward in their category.

FDA Grants Breakthrough Therapy Designation for BioMarin’s Valoctocogene Roxaparvovec (formerly BMN 270), an Investigational Gene Therapy for Hemophilia A

BioMarin announced in October that the U.S. Food and Drug Administration (FDA) granted valoctocogene roxaparvovec (formerly BMN 270) Breakthrough Therapy Designation.

BioMarin Provides 1.5 years of Clinical Data for Valoctocogene Roxaparvovec Gene Therapy for Severe Hemophilia A at 59th American Society of Hematology (ASH) Annual Meeting Concurrent with NEJM Publication

BioMarin announced in December an update to its previously reported results of an open-label Phase 1/2 study of valoctocogene roxaparvovec (formerly BMN 270), an investigational gene therapy treatment for severe hemophilia A.

BioMarin Highlights New Results for Gene Therapy Valoctocogene Roxaparvovec at the 2017 American Society of Hemophilia (ASH) Meeting

BioMarin announced in December updates on valoctocogene roxaparvovec (formerly BMN 270), an investigational gene therapy treatment for severe hemophilia at ASH.

BioMarin Doses First Patient in Global GENEr8-1 Phase 3 Study of Valoctocogene Roxaparvovec Gene Therapy for Severe Hemophilia A

BioMarin announced in December that it has dosed the first patient in the global GENEr8-1 Phase 3 study with the 6e13 vg/kg dose for valoctocogene roxaparvovec (formerly BMN 270), an investigational gene therapy for the treatment of patients with severe hemophilia A.

BioMarin Receives Anticipated Notification of PDUFA Extension for Pegvaliase Biologics License Application (BLA) to May 28, 2018

BioMarin announced in December that the U.S. Food and Drug Administration (FDA) will require additional time to complete its review of the Biologics License Application (BLA) for its investigational therapy pegvaliase, a PEGylated recombinant phenylalanine ammonia lyase enzyme product, to reduce blood phenylalanine (Phe) levels in adult patients with phenylketonuria (PKU) who have uncontrolled blood Phe levels on existing management.

2016

BioMarin Announces Addition of David Pyott, Former Allergan Chairman and CEO, to Company’s Board of Directors

BioMarin announced in January that David Pyott had been elected to the company’s board of directors. Mr. Pyott is the former Chairman and Chief Executive Officer of Allergan and joined the BioMarin Board immediately.

BioMarin Announces Interim Analysis of INSPIRE Clinical Trial in Pompe Disease at 34th Annual J.P. Morgan Annual Healthcare Conference

BioMarin announced in January interim results from INSPIRE, a Phase 2 trial for reveglucosidase alfa, a fusion protein of insulin-like growth factor 2 and acid alpha-glucosidase (IGF2-GAA) being studied for the treatment of late-onset Pompe disease (LOPD). The interim efficacy and safety analysis is based on 24 patients who previously had been on treatment with the enzyme replacement therapy, alglucosidase alfa, and were switched to reveglucosidase alfa.

FDA Issues Complete Response Letter for KyndrisaTM for Duchenne Muscular Dystrophy Amenable to Exon 51 Skipping

BioMarin announced in January that the U.S. Food and Drug Administration (FDA) issued a Complete Response letter to the Company’s New Drug Application (NDA) for KyndrisaTM (drisapersen) for the treatment of Duchenne muscular dystrophy (Duchenne) amenable to exon 51 skipping.

BioMarin Announces BioMarin RareConnections™ Rebranding and Consolidating BioMarin’s Comprehensive Support and Services for Eligible Rare Disease Patients

BioMarin announced in February the launch of BioMarin RareConnectionsTM, rebranding its patient-focused offering formally known as the BioMarin Patient and Physician Support (BPPS). For over 10 years, BioMarin has been a leader in designing and implementing multiple separate service offerings to support our patients. With the launch of BioMarin RareConnections, it now provides them under one expanded program, which includes support for eligible patients throughout treatment and assistance to gain and maintain access to BioMarin’s innovative therapies.

BioMarin Receives Orphan Drug Designation From FDA for First AAV-Factor VIII Gene Therapy, BMN 270, for Patients With Hemophilia A

BioMarin announced in March that BMN 270, an investigational gene therapy for the treatment of patients with hemophilia A, has been granted orphan drug designation by the U.S. Food and Drug Administration (FDA). BioMarin is currently conducting a Phase 1/2 study to evaluate the safety and efficacy of BMN 270 gene therapy in up to 12 patients with severe hemophilia A and will provide a program update in April. BMN 270 is an AAV 5 factor VIII vector, designed to restore factor VIII plasma concentrations, essential for blood clotting in patients with hemophilia A. The FDA Orphan Drug program provides orphan designation to drugs and biologics that are intended for the treatment of rare diseases (those affecting fewer than 200,000 people in the United States).

BioMarin Announces Positive Data From Cerliponase Alfa Program for Treatment of CLN2 Disease, a Form of Batten Disease, at 12th Annual WORLDSymposium(TM) 2016

BioMarin announced in March positive 48-week results from its Phase 1/2 pivotal study for cerliponase alfa, a recombinant human tripeptidyl peptidase 1 (rhTPP1) to treat children with CLN2 disease, a form of Batten disease. The average rate of clinical decline for motor and language function in patients receiving cerliponase alfa treatment — the primary efficacy endpoint — was approximately 80% less than the expected rate of decline in the untreated population, preserving essential function in the majority of treated patients (p <0.0001). Treatment with 300 mg cerliponase alfa administered via intracerebroventricular (ICV) infusion every other week was generally safe and well-tolerated in 24 patients and resulted in disease stabilization in 65% (15 of 23) of patients treated over a 48-week period, based on the Hamburg Motor + Language CLN2 rating. BioMarin estimates the incidence of CLN2 disease is approximately one in 200,000 with approximately 1,200 to 1,600 children in BioMarin's commercial territories.

BioMarin Phase 3 Study of Pegvaliase for Phenylketonuria (PKU) Meets Primary Endpoint of Blood Phenylalanine (Phe) Reduction (p<0.0001)

BioMarin announced in March that the pivotal Phase 3 PRISM-2 study (formerly referred to as 165-302) of pegvaliase met the primary endpoint of change in blood Phe compared with placebo (p<0.0001) in preliminary results. During the 8 week PRISM-2 double-blind, placebo-controlled, randomized drug discontinuation trial (RDT), 86 patients were randomized to either remain on pegvaliase or receive matching placebo. The pegvaliase treated group maintained mean blood Phe levels at 527.2 umol/L compared to their RDT baseline of 503.9 umol/L, whereas the placebo treated group mean blood Phe levels increased to 1385.7 umol/L compared to their RDT baseline of 536.0 umol/L . (see Table 1) The treatment effect demonstrated in this study represents an approximately 62% improvement in blood Phe compared to placebo.

BioMarin Receives European Orphan Drug Designation for BMN 270, First Investigational AAV-Factor VIII Gene Therapy for Patients with Hemophilia A

BioMarin announced in March that BMN 270, an investigational gene therapy for the treatment of hemophilia A, has been granted orphan drug designation by the European Commission. In the European Union, orphan drug designation is given to treatments that are intended for life-threatening or chronically-debilitating conditions with a prevalence of not more than 5 in 10,000 people. Earlier this month, BioMarin announced BMN 270 had also received orphan drug designation from the U.S. Food and Drug Administration.

BioMarin Provides Encouraging Preliminary Data on First 8 Patients in Hemophilia A Gene Therapy Program

BioMarin announced in April preliminary data from an ongoing Phase 1/2 clinical trial with BMN 270, an investigational gene therapy treatment for hemophilia A. A total of eight patients with severe hemophilia A received a single dose of BMN 270, six of whom have been treated at the highest dose of 6 x 1013 vg/kg, and to date, post-treatment follow-up ranges from five to 16 weeks. At last observation, patients at the highest dose experienced increasing Factor VIII activity levels ranging between 4% and 60% (as a percentage calculated based on the numbers of International Units (IU) per milliliter of whole blood), with five of six patients treated at the high dose now over 5% and two of six at over 50%. All high dose patients improved from severe to either moderate, mild or normal range in terms of factor levels based on World Federation of Hemophilia criteria.

BioMarin Enrolls First Patient in Phase 1/2 Trial of NAGLU Fusion Protein BMN 250 for Treatment of MPS IIIB (Sanfilippo B Syndrome)

BioMarin announced in April that it had enrolled the first patient in a Phase 1/2 trial for BMN 250, an investigational enzyme replacement therapy using a novel fusion of recombinant human alpha-N-acetyglucosaminidase (NAGLU) with a peptide derived from insulin-like growth factor 2 (IGF2), for the treatment of Sanfilippo B syndrome or mucopolysaccharidosis IIIB (MPS IIIB). Discovered by BioMarin, BMN 250 is being studied in a multicenter, international clinical trial evaluating safety and tolerability, as well as cognitive function of patients with MPS IIIB receiving BMN 250. Designed to restore functional NAGLU activity in the brain, BMN 250 is administered via intracerebroventricular (ICV) infusion.

BioMarin Announces EMA Grants Accelerated Assessment for Cerliponase Alfa, Experimental Treatment for a Form of Batten Disease

BioMarin announced in May that the European Medicines Agency (EMA) has granted BioMarin’s request for accelerated assessment for the planned cerliponase alfa Marketing Authorization Application (MAA). Accelerated assessments are granted on the grounds that a product may satisfy an unmet medical need and is of major interest from the point of view of therapeutic innovation and public health. Accelerated assessment has the potential to shorten EMA’s review procedure. However, at any time during the MAA assessment, the EMA may decide to continue the assessment under standard assessment timelines, and most applications that initially qualify for accelerated assessment are ultimately reviewed on a standard timeline.

BioMarin Announces Withdrawal of Market Authorization Application for Kyndrisa™ (drisapersen) in Europe

BioMarin announced in May that it has withdrawn its Kyndrisa™ (drisapersen) Marketing Authorization Application (MAA) from the European Medicines Agency (EMA) following discussions at the May 2016 Committee for Medicinal Products for Human Use (CHMP) meeting. Those discussions clearly indicated that the CHMP intended to issue a negative opinion. Kyndrisa is an experimental drug for the treatment of Duchenne muscular dystrophy (DMD) amenable to exon 51 skipping.

BioMarin Appoints Two BioPharmaceutical Veterans to Board of Directors, Willard Dere, M.D. and Kathryn E. Falberg

BioMarin announced in July the appointment of two biopharmaceutical veterans to its Board of Directors: Willard Dere, M.D., Professor of Internal Medicine at the University of Utah Health Sciences Center and former Senior Vice President in Research and Development at Amgen and Kathryn E. Falberg, former Chief Financial Officer of Jazz Pharmaceuticals and Amgen.

BioMarin Provides Positive Proof-of-Concept Data for BMN 270 Gene Therapy in Hemophilia A in Late Breaking Oral Presentation at the World Federation of Hemophilia (WFH) 2016 World Congress

BioMarin announced in July positive interim results of an open-label Phase 1/2 study of BMN 270, an investigational gene therapy treatment for severe hemophilia A at the XXXII International Congress of the World Federation of Hemophilia (WFH). The data was presented in the Late Breaking Gene Therapy session by John Pasi, Professor of Haemostasis and Thrombosis, Barts and the London School of Medicine, Honorary Consultant Haematologist, The Royal London Hospital, and a lead investigator of the study.

FDA Accepts BLA for BioMarin’s Cerliponase Alfa for CLN2 Disease, Form of Batten Disease

BioMarin announced in July that the U.S. Food and Drug Administration (FDA) accepted for review the submission of a Biologics License Application (BLA) for cerliponase alfa, an investigational therapy to treat children with CLN2 disease, a form of Batten disease. The Prescription Drug User Fee Act (PDUFA) goal date for a decision is January 27, 2017. BioMarin also has submitted a Marketing Authorization Application (MAA) to the European Medicines Agency (EMA) for cerliponase alfa, and it is undergoing validation at the Agency.

BioMarin Announces Pricing of Public Offering of Common Stock

BioMarin announced in August the pricing of an underwritten public offering of 7,500,000 shares of its common stock at a price to the public of $96.00 per share. The gross proceeds to BioMarin from this offering are expected to be approximately $720.0 million, before deducting underwriting discounts and commissions and estimated offering expenses payable by BioMarin.

Forbes Ranks BioMarin 10th Most Innovative Company in the World

BioMarin announced in August that it has been ranked 10th on Forbes magazine’s 2016 list of the “World’s Most Innovative Companies.” This is the third consecutive year that BioMarin has received the recognition, acknowledging the company’s ongoing commitment to rapidly developing and delivering first-in-class or best-in-class therapies for those with severe or life-threatening rare diseases. Last year, the company was also ranked 10th, and in 2014, the company ranked 7th.

BioMarin Announces Update to Brineura™ (Cerliponase Alfa) Program for Treatment of CLN2 Disease, a Form of Batten Disease

BioMarin announced in September a program update for cerliponase alfa, a recombinant human tripeptidyl peptidase 1 (rhTPP1) to treat children with CLN2 disease, a form of Batten disease.

BioMarin Announces EMA Validation of Brineura™ (Cerliponase Alfa) Marketing Authorization Application for Treatment of CLN2 Disease, a Form of Batten Disease

BioMarin announced in September that the European Medicines Agency (EMA) validated the Marketing Authorization Application (MAA) for Brineura™ (cerliponase alfa) to treat children with CLN2 disease, a form of Batten disease. Validation of the MAA confirms that the submission is accepted and starts the formal review process by the EMA’s Committee for Human Medicinal Products (CHMP).

BioMarin Reviews Status of Exon 51 Composition of Matter and Method of Use Patent Interference Cases against Sarepta Therapeutics

BioMarin announced in September that it intends to seek a review of the Patent Trial and Appeal Board (PTAB) of the United States ruling in Interference No. 106,008, related to composition of matter (COM) claims related to exon 51 skipping antisense oligonucleotides. BioMarin is completing its review of the decision and the specific means it may use to seek a further review.

UK Regulatory Agency Approves Continued Enrollment in BioMarin Phase 1/2 Study of BMN 270 in Hemophilia A

BioMarin announced in October that the Medicines and Healthcare Products Regulatory Agency (MHRA) in the United Kingdom approved continued enrollment into the open-label Phase 1/2 study of BMN 270, an investigational gene therapy treatment for severe hemophilia A.

BioMarin Presents Vosoritide Data in Achondroplasia at American Society of Human Genetics (ASHG) 2016 Meeting

BioMarin provided an update on its Phase 2 study of vosoritide, an analog of C-type Natriuretic Peptide (CNP), in children with achondroplasia, the most common form of dwarfism, at the American Society of Human Genetics 2016 Meeting. Results from 8 children in cohort 4, who completed six months of daily dosing at 30 µg/kg/daily experienced a 46% or 2.1 cm/year increase in mean annualized growth velocity from baseline (p-value = 0.03). These data are comparable to those observed at the lower dose of 15 µg/kg/day in cohort 3. Results from 10 children in cohort 3, who completed six months of daily dosing at 15 µg/kg/day experienced a 50% or 2.0 cm/year increase in mean annualized growth velocity from baseline (p-value = 0.01).

BioMarin Enrolls First Participant in Phase 3 Trial of Vosoritide for Treatment of Children with Achondroplasia

BioMarin announced in December that the company has initiated a global Phase 3 study for vosoritide, an analog of C-type Natriuretic Peptide (CNP), in children with achondroplasia, the most common form of dwarfism. The first child enrolled in the study was at a site in Australia.

2015

Ernst & Young Names BioMarin CEO Jean-Jacques Bienaime EY Entrepreneur Of The Year® 2015 National Award Winner in Life Sciences

Ernst & Young Global Limited in November awarded BioMarin CEO Jean-Jacques Bienaimé the EY Entrepreneur Of The Year® 2015 National Award in the Life Sciences category. The award recognizes outstanding entrepreneurs who demonstrate excellence and extraordinary success in such areas as innovation, financial performance, and personal commitment to their businesses and communities.

BioMarin’s Initial 6-Month Data from Phase 2 Study of Vosoritide (BMN 111) in Children with Achondroplasia Presented at the American Society for Bone and Mineral Research Annual 2015 Meeting

Dr. Melita Irving, Clinical Geneticist, Guy’s and St Thomas’ NHS Foundation Trust, Evelina Children’s Hospital London, UK, in October presented the initial six-month data from the first three cohorts of BioMarin’s Phase 2 proof-of-concept and dose-finding study of vosoritide (BMN 111), an analog of C-type Natriuretic Peptide, in children with achondroplasia at the American Society for Bone and Mineral Research Annual 2015 Meeting in Seattle, Washington. Achondroplasia is the most common form of human dwarfism. The initial six-month data from the first three cohorts showed a 50 percent or 2.01 cm/year increase in mean annualized growth velocity (speed at which growth in children occurs) in the cohort of 10 patients receiving a 15 µg/kg dose of vosoritide daily for six months compared with their own pre-treatment growth velocity (p-value= 0.01).

BioMarin to Acquire Rights to Phenylketonuria (PKU) Franchise From Merck Serono

BioMarin in October announced that it acquired all global rights to Kuvan® (sapropterin dihydrochloride) and pegvaliase from Merck Serono (Merck). Under the terms of the agreement, BioMarin will provide Merck with an upfront payment of €340 million. An additional €60 million in milestones will be paid to Merck if combined sales of Kuvan and pegvaliase reach undisclosed cumulative sales thresholds. In addition, €125 million will be paid to Merck for regulatory milestones related to pegvaliase. BioMarin will now have exclusive worldwide rights to Kuvan and pegvaliase with the exception of Kuvan in Japan.

BioMarin Enrolls First Patient in Phase 1/2 Trial of Gene Therapy Drug Candidate BMN 270 for the Treatment of Hemophilia A

BioMarin in September announced that it has enrolled the first patient in a Phase 1/2 trial for BMN 270, an investigational gene therapy for the treatment of patients with hemophilia A. BMN 270 is an AAV-factor VIII vector, designed to restore factor VIII plasma concentrations, essential for blood clotting in patients with hemophilia A.

Forbes Ranks BioMarin 10th Most Innovative Company in the World

Forbes magazine in August ranked BioMarin 10th on its 2015 list of the “World’s Most Innovative Companies.” This is the second consecutive year that BioMarin has received the recognition, reinforcing the company’s promise to deliver new therapeutics to patients with severe or life-threatening diseases. The company was previously ranked seventh.

BioMarin Joins the NASDAQ-100 Index

BioMarin in July announced that it will become a component of the NASDAQ-100 Index® (NASDAQ:NDX), the NASDAQ-100 Equal Weighted Index (NASDAQ:NDXE) and the NASDAQ-100 Ex-Technology Index (NASDAQ:NDXX) prior to market open on Monday, July 27, 2015. The NASDAQ-100 Index is composed of the 100 largest non-financial stocks listed on The NASDAQ Stock Market based on market capitalization.

BioMarin Announces EMA Validates MAA for Drisapersen for Treatment of Duchenne Muscular Dystrophy Amenable to Exon 51 Skipping

The European Medicines Agency (EMA) in June validated the Marketing Authorization Application (MAA) for BioMarin’s drisapersen for the treatment of Duchenne Muscular Dystrophy amenable to exon 51 skipping. Validation of the MAA confirms that the submission is complete and starts the EMA’s standard review process. Day 120 questions will be received on 22 October 2015, leading to a potential CHMP opinion in the first half of 2016 and a European Commission Decision by the third quarter of 2016.

BMN 111 (vosoritide) Improves Growth Velocity in Children With Achondroplasia in Phase 2 Study

BioMarin in June announced positive results of a Phase 2 proof-of-concept and dose finding study of BMN 111 (vosoritide), an analog of C-type Natriuretic Peptide (CNP), in children with achondroplasia. Achondroplasia is the most common form of human dwarfism. Vosoritide has Orphan designation in both the United States and Europe.

BioMarin CEO Jean-Jacques Bienaime Awarded EY Entrepreneur Of The Year(R) 2015 Award in Northern California in Health and Life Sciences

EY in June awarded BioMarin CEO Jean-Jacques Bienaimé the EY Entrepreneur Of The Year® 2015 Award in the Health and Life Sciences category in Northern California. The award recognizes outstanding entrepreneurs who demonstrate excellence and extraordinary success in such areas as innovation, financial performance, and personal commitment to their businesses and communities.

BioMarin Announces Completion of Tender Offer for Prosensa’s Shares and Commences Subsequent Offering Period

BioMarin in January announced it completed the initial offering period for the previously announced tender offer for all of the outstanding ordinary shares of Prosensa Holding N.V. (Nasdaq:RNA). As a result, BioMarin will purchase approximately 93.4% of Prosensa’s outstanding shares pursuant to the initial offering period for the tender offer. BioMarin also announced that it has commenced a subsequent offering period to provide Prosensa shareholders who have not yet tendered their shares the opportunity to do so. The subsequent offering period is scheduled to expire at 6:00 p.m., New York City time, on January 29, 2015.

BioMarin Provides Preliminary Data From Ongoing Phase 1/2 Pivotal Study of BMN 190 for Treatment of CLN2 Disorder, a Form of Batten Disease

BioMarin in January announced interim results from its Phase 1/2 pivotal study for BMN 190 or cerliponase alfa, a recombinant human tripeptidyl peptidase 1 (rhTPP1), to treat of patients with late infantile CLN2 disease, a form of Batten disease. Interim data indicates that in all nine of the BMN 190 patients who have been followed for at least six months and up to 15 months, the treatment appears to show stabilization of the disease compared to the natural history based on a standardized measure of motor and language function.

2014

BioMarin Announces Approval of Vimizim® (elosulfase alfa) in Japan for Treatment of Morquio A Syndrome

Japan’s Ministry of Health, Labor and Welfare granted approval of the registration of BioMarin’s Vimizim® (elosulfase alfa) for the treatment of patients with mucopolysaccharidosis type IVA (MPS IVA), also known as Morquio A syndrome. Vimizim is the first treatment in Japan approved for this condition. Vimizim was reviewed under the Orphan Drug program.

BioMarin’s Vimizim® (elosulfase alfa) Approved in Brazil for Treatment of Morquio A Syndrome

The Agência Nacional de Vigilancia Sanitaria (ANVISA), or the National Health Surveillance Agency Brazil, in December approved BioMarin’s Vimizim® (elosulfase alfa) for the treatment of patients with mucopolysaccharidosis type IVA (MPS IVA), also known as Morquio A syndrome. Vimizim is the first and only treatment for the ultra-rare genetic condition.

FDA Grants BioMarin Orphan Drug Designation for NAGLU Fusion Protein, Tralesinidase alfa (BMN 250), for MPS IIIB

The Food and Drug Administration in December granted orphan drug designation for Tralesinidase alfa (BMN 250), a novel fusion of alpha-N-acetyglucosaminidase (NAGLU) with a peptide derived from insulin-like growth factor 2 (IGF2), for the treatment of Sanfilippo Syndrome Type B or Mucopolysaccharidosis IIIB (MPS IIIB). Tralesinidase alfa (BMN 250) is an enzyme replacement therapy using recombinant human NAGLU with an IGF2, or Glycosylation Independent Lysosomal Targeting (GILT) tag. Tralesinidase alfa (BMRN 250) is delivered directly to the brain using BioMarin’s patented technology.

BioMarin’s Vimizim® (elosulfase alfa) Approved in Australia for Treatment of Morquio A Syndrome

In December, the Australian Therapeutic Goods Administration granted approval of the registration of Vimizim® (elosulfase alfa) for the treatment of patients with mucopolysaccharidosis type IVA (MPS IVA), also known as Morquio A syndrome. Vimizim is the first treatment in Australia approved for this condition.

BioMarin Agrees to Acquire Prosensa Holding N.V.

In November, BioMarin entered a definitive agreement to offer to purchase all of the outstanding ordinary shares of Prosensa Holding N.V. for $17.75 per share, for a total up front consideration of approximately $680 million. In addition, two approximately $80 million contingent milestones are payable for the approval of drisapersen, Prosensa’s drug candidate in development for certain forms of Duchenne muscular dystrophy in the U.S. no later than May 15, 2016 and Europe no later than February 15, 2017, respectively.

BioMarin Named by CenterWatch as One of the Fastest Developers of Medicines

In September, CenterWatch, a leading source for global clinical trial information, named BioMarin as one of the fastest drug developers in the industry.

BioMarin Sells Priority Review Voucher for $67.5 Million

In July, BioMarin announced that it has sold the Rare Pediatric Disease Priority Review Voucher it obtained when it won approval of Vimizim in February under an FDA program intended to encourage the development of treatments for rare pediatric diseases, for $67.5 million to Regeneron Ireland, an indirect, wholly-owned subsidiary of Regeneron Pharmaceuticals, Inc.

BioMarin Announces Health Canada Approval of Vimizim™ (elosulfase alfa) for the Treatment of Morquio A Syndrome

Health Canada in July approved Vimizim® (elosulfase alfa) for long-term enzyme replacement therapy in patients with a confirmed diagnosis of mucopolysaccharidosis IVA (MPS IVA), also known as Morquio A syndrome. The approval makes Vimizim the first and only pharmaceutical treatment option available in Canada for children and adults living with this severely debilitating, progressive and life-limiting disorder.

BioMarin Doses First Patient in Phase 3 INSPIRE Trial With BMN 701 for the Treatment of Pompe Disease

BioMarin in May announced today that it has dosed the first patient with BMN 701 (GILT-tagged Recombinant Human GAA) in the Phase 3 INSPIRE trial for Pompe disease. BMN 701 is a novel fusion protein of insulin-like growth factor 2 and acid alpha glucosidase (IGF2-GAA), designed to target delivery to the lysosomes where the enzyme is most needed.

BioMarin Announces European Commission Approval for Vimizim(R) (elosulfase alfa) for the Treatment of Morquio A Syndrome in Patients of All Ages

The European Commission in April granted marketing authorization for Vimizim® (elosulfase alfa), the first specific treatment approved in the European Union for Mucopolysaccharidosis type IVA (MPS IVA), also known as Morquio A syndrome, in patients of all ages. As the first drug ever approved for Morquio A syndrome, Vimizim has been granted orphan drug status in the European Union, which confers ten years of market exclusivity.

BioMarin Appoints Pioneer in Personalized Medicine, Dennis J. Slamon, M.D., Ph.D., to Board of Directors

BioMarin in March announced the appointment of Dennis J. Slamon, M.D., Ph.D., director of Clinical/Translational Research and director of the Revlon/UCLA Women’s Cancer Research Program at UCLA’s Jonsson Comprehensive Cancer Center, to the company’s board of directors.

BioMarin Announces Pricing of Public Offering of Common Stock

BioMarin announced today the pricing on March 4, 2014 of an underwritten public offering of 1.5 million shares of its common stock. The gross proceeds to BioMarin from this offering were expected to be approximately $119.3 million before deducting underwriting discounts and commissions and estimated offering expenses payable by BioMarin.

BioMarin Announces FDA Approval for Vimizim(TM) (elosulfase alfa) for the Treatment of Patients With Morquio A Syndrome

The U.S. Food and Drug Administration in February approved Vimizim™ (elosulfase alfa) for patients with Mucopolysaccharidosis type IVA (MPS IVA), also known as Morquio A syndrome.

BioMarin Announces Agreement With Repligen for Pre-clinical Compounds

BioMarin announced in January that it has entered into an agreement to purchase Repligen Corporation’s histone deacetylase inhibitor (HDACi) library and related intellectual property. Potential applications of the HDACi portfolio include Friedreich’s ataxia and other neurological disorders.

BioMarin Doses First Patient in Phase 2 Trial With BMN 111 for the Treatment of Children With Achondroplasia

BioMarin in January announced today that it had dosed the first child in the Phase 2 trial with BMN 111, an analog of C-type Natriuretic Peptide (CNP), for the treatment of children with achondroplasia. Achondroplasia is the most common form of disproportionate short stature or dwarfism.

BioMarin Announces Selection of Factor VIII Gene Therapy Drug Development Candidate BMN 270 for the Treatment of Hemophilia A

BioMarin announced in January that it has selected an AAV-factor VIII vector, BMN 270, to develop for the treatment of hemophilia A and has initiated IND-enabling studies.

2013

FDA Advisory Committee Recommends Approval for BioMarin’s Vimizim™ for the Treatment of Patients with Morquio A Syndrome

In November, BioMarin announced that the Endocrinologic and Metabolic Drugs Advisory Committee (EMDAC) of the U.S. Food and Drug Administration (FDA) voted in favor of approval of Vimizim for the treatment of Morquio A syndrome, also called Mucopolysaccharidosis Type IVA (MPS IVA).

BioMarin Announces French ATU Granted for Vimizim™ for the Treatment of Morquio A Syndrome

In November, BioMarin announced that the French National Agency for Medicines and Health Products Safety (ANSM) granted an Autorisation Temporaire d’Utilisation de cohorte (ATU cohort), or Temporary Authorization for Use, for patient sales of Vimizim for the treatment of Morquio A Syndrome. An ATU is the regulatory mechanism used by the ANSM to make non-approved drugs available to patients in France when a genuine public health need exists.

BioMarin Doses First Patient in Phase 1/2 Trial with BMN 190 for the Treatment of Neuronal Ceroid Lipofuscinosis Type 2, a Form of Batten Disease

In September, BioMarin announced that it dosed the first patient in the Phase 1/2 trial for BMN 190, a recombinant human tripeptidyl peptidase 1 (rhTPP1) for the treatment of patients with neuronal ceroid lipofuscinosis type 2 (NCL-2), a form of Batten disease. This is the first time that a patient with Batten disease has been treated with an enzyme replacement therapy in a clinical trial setting.

BioMarin Appoints Richard Ranieri as Senior Vice President, Human Resources and Corporate Affairs

In September, BioMarin announced the appointment of Richard Ranieri as Senior Vice President, Human Resources and Corporate Affairs.

BioMarin Initiates Phase 3 Trial for PEG-PAL for the Treatment of PKU

In June, BioMarin announced that it initiated the Phase 3 program for PEG-PAL (PEGylated recombinant Phenylalanine Ammonia Lyase) for the treatment of phenylketonuria (PKU).

FDA Accepts Vimizim BLA and Grants Priority Review Designation

In May, BioMarin announced that the U.S. Food and Drug Administration (FDA) accepted for review the Biologics License Application (BLA) for Vimizim (BMN-110, elosulfase alfa), an enzyme replacement therapy under evaluation for the treatment of patients with the rare lysosomal storage disorder Mucopolysaccharidosis Type IVA (MPS IVA), also called Morquio A Syndrome.

Vimizim MAA Validated by the EMA

In May, BioMarin announced that the European Medicines Agency (EMA) validated the Marketing Authorization Application (MAA) for Vimizim. Validation of the MAA confirms that the submission is complete and starts the EMA’s formal review process.

BioMarin Advances BMN-701 for Pompe Disease to Next Phase of Development

In March, BioMarin announced results from POM-001, the Phase 1/2 trial for BMN-701, a fusion protein of insulin-like growth factor 2 and acid alpha-glucosidase (IGF2-GAA) for the treatment of late-onset Pompe disease.

BioMarin Licenses Factor VIII Gene Therapy Program for Hemophilia A From University College London and St. Jude Children’s Research Hospital

In February, BioMarin announced that it had licensed a Factor VIII gene therapy program for hemophilia A from University College London (UCL) and St. Jude Children’s Research Hospital.

BioMarin Announces Kuvan Significantly Improves Inattentiveness in Kuvan Responding PKU Patients

In February, BioMarin announced results from the PKU-016 ASCEND study, the largest randomized controlled trial evaluating neuropsychiatric outcomes in phenylketonuria (PKU) patients treated with the approved drug Kuvan (sapropterin dihydrochloride).

BioMarin Acquires Zacharon Pharmaceuticals

In January, BioMarin announced that it acquired Zacharon Pharmaceuticals, a private biotechnology company based in San Diego, focused on developing small molecules targeting pathways of glycan and glycolipid metabolism.

2012

BioMarin Phase 3 Study of GALNS for the Treatment of MPS IVA Meets Primary Endpoint

In November, BioMarin announced that the pivotal Phase 3 study of GALNS met the primary endpoint of change in six-minute walk distance compared with placebo at 24 weeks in subjects receiving weekly infusions of GALNS at the dose of 2 mg/kg.

BioMarin Announces Decision to Start Phase 3 Program for PEG-PAL

In September, BioMarin announced preliminary results from the Phase 2 program of PEG-PAL (PEGylated recombinant Phenylalanine Ammonia Lyase) for the treatment of phenylketonuria (PKU) demonstrating long-term retention, tolerability and providing evidence of efficacy. Based on these results, the company expects to start a pivotal Phase 3 study in the second quarter of 2013, following an anticipated end of Phase 2 meeting with the FDA in the first quarter of 2013.

BioMarin Announces Phase 1 Results for BMN-111 for Achondroplasia

In September, BioMarin announced the completion of a Phase 1 study for BMN-111, an analog of C-type Natriuretic Peptide (CNP), for achondroplasia.

BioMarin Names Jeff Ajer Senior Vice President, Chief Commercial Officer

In September, BioMarin announced that Jeff Ajer, the previous Vice President, Commercial Operations, the Americas, was promoted to Senior Vice President, Chief Commercial Officer.

BioMarin Appoints Dan Spiegelman as Executive Vice President and Chief Financial Officer

In May, BioMarin announced the appointment of Dan Spiegelman as Executive Vice President and Chief Financial Officer (CFO).

BioMarin Completes Enrollment for Phase 3 Trial for GALNS for the Treatment of MPS IVA

In March, BioMarin announced that enrollment was complete for the pivotal Phase 3 trial for N-acetylgalactosamine 6-sulfatase (GALNS or BMN-110), intended for the treatment of the lysosomal storage disorder Mucopolysaccharidosis Type IVA (MPS IVA), also called Morquio A Syndrome.

BioMarin Initiates Phase 1 Trial for BMN-111 for the Treatment of Achondroplasia

In February, BioMarin announced the initiation of a Phase 1 study in healthy volunteers for BMN-111, an analog of C-type Natriuretic Peptide (CNP), for the treatment of achondroplasia.

2011

BioMarin Receives Positive Opinion from European Regulatory Authorities for Expanded Biologics Manufacturing Facility

In December, BioMarin announced that the Committee for Medicinal Products for Human Use (CHMP) of the European Medicines Agency (EMA) has recommended approval for the company’s manufacturing facility expansion in Novato, CA.

BioMarin Announces BMN-190 for Late infantile neuronal ceroid lipofuscinosis (LINCL) – Form of Batten Disease

In December, at its R&D Day, BioMarin announced a new clinical program, BMN-190 for LINCL, one form of Batten disease.

BioMarin Announces Buy Back of Naglazyme Royalties From Adelaide Health Authority

In November, BioMarin completed the buy back of certain intellectual property from SA Pathology, a unit of the Central Adelaide Local Health Network located in Adelaide, Australia for an upfront payment of $81 million. The intellectual property includes patents related to the purified form of Naglazyme and the method of using the enzyme in the treatment of MPS VI, which expire between 2022 and 2023.

BioMarin Initiates Phase 2 Study for GALNS in Patients Under Five Years of Age With MPS IVA

In November, BioMarin initiated a Phase 2 study for GALNS (N-acetylgalactosamine 6-sulfatase) in patients under five years of age with mucopolysaccharidosis IVA (MPS IVA).

BioMarin Announces FDA Approval of Expanded Biologics Manufacturing Facility

In November, BioMarin announced tthat it had received approval from the U.S. Food and Drug Administration (FDA) for its manufacturing facility expansion in Novato, CA.

BioMarin Agrees to Acquire Biologics Manufacturing Plant in Ireland from Pfizer

In July, BioMarin announced that it had entered into a definitive agreement to acquire a bulk biologics manufacturing plant from Pfizer, located in Shanbally, Cork, Ireland. The transaction was completed in August of 2011.

BioMarin Initiates Phase 3 Trial for Amifampridine Phosphate for the Treatment of LEMS

In June, BioMarin initiated a Phase 3 trial for amifampridine phosphate (3,4-diaminopyridine phosphate) for the treatment of patients with Lambert-Eaton Myasthenic Syndrome (LEMS).

BioMarin Initiates Pivotal Phase 3 Trial for GALNS for the Treatment of MPS IVA

In February, BioMarin initiated a pivotal Phase 3 trial for N-acetylgalactosamine 6-sulfatase (GALNS or BMN 110), intended for the treatment of the lysosomal storage disorder Mucopolysaccharidosis Type IVA (MPS IVA), also called Morquio A Syndrome.

BioMarin Initiates Phase 1/2 Trial for BMN 701 for the Treatment of Pompe Disease

In January, BioMarin initiated a Phase 1/2 trial for BMN 701, a novel fusion protein of insulin-like growth factor 2 and acid alpha glucosidase (IGF2-GAA) in development for the treatment of Pompe disease.

2010

BioMarin Announces Program for BMN-111 for the Treatment of Achondroplasia

In October 2010, BioMarin announced its program for BMN-111, a peptide therapeutic for the treatment of achondroplasia. BioMarin plans to file an IND in the fourth quarter of 2011 and to initiate a Phase 1 clinical trial by the first quarter of 2012.

BioMarin Appoints William Young and Kenneth Bate to Its Board of Directors

In September 2010, BioMarin announced the appointment of William D. Young and Kenneth M. Bate to its Board of Directors.

BioMarin Receives Orphan Drug Designation from the FDA for BMN-701 for the
Treatment of Pompe Disease

In August 2010, BioMarin received orphan drug designation from the U.S. Food and Drug Administration (FDA) for BMN-701, a novel fusion of insulin-like growth factor 2 and alpha glucosidase (IGF2-GAA) in development for the treatmen of Pompe disease.

BioMarin Acquires ZyStor Therapeutics, Inc.

In August 2010, BioMarin acquired ZyStor Therapeutics, Inc., a privately-held biotechnology company developing enzyme replacement therapies (ERT) for the treatment of lysosomal storage disorders.

BioMarin Initiates Phase 3b Study to Evaluate the Effects of Kuvan on Neurophychiatric
Symptoms in Subjects with PKU

In August 2010, BioMarin announced that the first subject has initiated treatment in a Phase 3b study (PKU-016) to evaluate the effects of Kuvan (sapropterin dihydrochloride) on neuropsychiatric symptoms in subjects with phenylketonuria (PKU).

BioMarin Halts Development of BMN-195 for Duchenne Muscular Dystrophy

In August 2010, BioMarin completed the Phase 1 clinical study of BMN 195, a small molecule utrophin up-regulator, for the treatment of Duchenne muscular dystrophy (DMD). The Phase 1 clinical trial results were not positive and the BMN 195 program was discontinued due to pharmaceutical and pharmacokinetic challenges.

BioMarin Reports Positive Results for Phase I/II Trial for BMN-110 for MPS
IVA

In April 2010, BioMarin announced positive results for the Phase I/II trial for BMN 110 or N-acetylgalactosamine 6-sulfatase (GALNS), intended for the treatment of the lysosomal storage disorder Mucopolysaccharidosis Type IVA (MPS IVA), or Morquio A Syndrome.

BioMarin Launches Firdapse in the European Union

In April 2010 BioMarin  launched Firdapse(TM) (3,4-diaminopyridine) in
the European Union (E.U.) for the treatment of the rare autoimmune disease Lambert Eaton Myasthenic Syndrome (LEMS).

BioMarin Acquires LEAD Therapeutics

In February 2010, BioMarin entered into a stock purchase agreement to acquire LEAD Therapeutics, Inc. (LEAD), a small private drug discovery and early stage development company with key compound LT-673, an orally available poly (ADP-ribose) polymerase (PARP) inhibitor for the treatment of patients with rare, genetically defined cancers.

BioMarin Initiates Phase 1 Clinical Study of BMN-195 for Duchenne Muscular
Dystrophy

In January 2010, BioMarin announced that the first subject had initiated treatment in the Phase 1 clinical study of BMN 195, a small molecule utrophin upregulator, for the treatment of Duchenne muscular dystrohpy (DMD).

Firdapse Receives Marketing Approval in the EU for LEMS

In January 2010, BioMarin announced today that the European Commission had granted marketing approval for Firdapse (3,4-diaminopyridine (amifampridine phosphate), for the rare autoimmune disease Lambert Eaton Myasthenic Syndrome (LEMS).

 

2009

FDA Grants Orphan Drug Designation for 3, 4-DAP for LEMS

In November 2009, the Food and Drug Administration (FDA) granted orphan drug designation for 3,4-diaminopyridine (3,4-DAP), amifampridine phosphate, for the rare autoimmune disease Lambert Eaton Myasthenic Syndrome (LEMS). 3,4-DAP has previously received orphan drug designation in the E.U. If approved by the European Commission, amifampridine phosphate will be the first approved treatment for LEMS in Europe.

BioMarin Acquires Huxley Pharmaceuticals, Inc.

In October 2009, the company acquired Huxley Pharmaceuticals, Inc., which has rights to a proprietary form of 3,4-diaminopyridine (3,4-DAP), amifampridine phosphate, for the rare autoimmune disease Lambert Eaton Myasthenic Syndrome (LEMS). The company expects to launch the product in Europe in the first quarter of 2010, and is evaluating a development strategy for amifampridine phosphate in LEMS in the U.S. and for other indications in the U.S. and Europe.

BioMarin Initiates Phase II Clinical Study of PEG-PAL in PKU

In September 2009, the company initiated the Phase II clinical study of PEG-PAL (PEGylated recombinant phenylalanine ammonia lyase) for the treatment of phenylketonuria (PKU). Initial top-line results are expected in mid-2010. The primary objective is to evaluate the effect of PEG-PAL on blood Phe concentrations in subjects with PKU. The secondary objectives are to evaluate the safety and tolerability, immune response and steady state pharmacokinetics of subcutaneous injections of multiple dose levels of PEG-PAL.

Kuvan Receives Priority Review Status From Health Canada

In June 2009, Kuvan® (sapropterin dihydrochloride) received priority review status from Health Canada. Priority review provides for a shortened submission review of 180 days versus the standard twelve months. BioMarin plans to file a marketing application for Kuvan in Canada in the third quarter of 2009, and with priority review status, a decision for marketing approval is expected in the first half of 2010.

BioMarin Initiates Phase I/II Clinical Trial for GALNS for Morquio A Syndrome (MPS IVA)

In April 2009, BioMarin initiated a Phase 1/2 clinical trial for BMN-110 or N-acetylgalactosamine 6-sulfatase (GALNS), intended for the treatment of the lysosomal storage disorder Mucopolysaccharidosis Type IVA (MPS IVA), or Morquio A Syndrome. The company expects to report initial results in the first half of 2010. The objectives of the Phase 1/2 study will be to evaluate safety, pharmacokinetics, pharmacodynamics and to identify the optimal dose of GALNS for future studies.

BioMarin Receives Notice of Allowance for Once Daily Dosing Patent for Kuvan

In March 2009, BioMarin received a notice from the U.S. Patent Office reporting allowance of claims covering once daily dosing methods for
Kuvan® (sapropterin dihydrochloride) Tablets
in the treatment of phenylketonuria (PKU). The company expects that the patent will be issued later in 2009. If issued, the patent’s 20-year term would expire in 2024. The company has a number of other pending patent applications covering various aspects of Kuvan compositions and dosing.

BioMarin’s Clinical Trial Application for GALNS for Morquio A Syndrome Accepted by the MHRA

In March 2009, BioMarin received formal acceptance from the United Kingdom Medicines and Healthcare Products Regulatory Agency (MHRA) for its application for the clinical trial authorization (CTA) for BMN 110 or N-acetylgalactosamine 6-sulfatase (GALNS), intended for the treatment of the lysosomal storage disorder Mucopolysaccharidosis Type IVA (MPS Type IVA) or Morquio A Syndrome. A Phase 1/2 clinical trial is expected to begin soon.

BioMarin Appoints Dr. Henry J. Fuchs as Senior Vice President and Chief Medical Officer

In March 2009, BioMarin welcomed Dr. Henry J. Fuchs as Senior Vice President and Chief Medical Officer (CMO). Dr. Fuchs replaced longtime BioMarin CMO, Dr. Emil Kakkis.

Naglazyme Approved by Brazil’s National Health Surveillance Agency

In March 2009, BioMarin received marketing approval from ANVISA, Brazil’s National Health Surveillance Agency, for Naglazyme® (galsulfase) for the treatment of patients with Mucopolysaccharidosis VI (MPS VI). Estimates indicate that Brazil has the largest known number of affected MPS VI patients in the world.

Results From Phase 2 Clinical Study of 6R-BH4 in Peripheral Arterial Disease Not Statistically Significant

In February 2009, BioMarin announced results from its Phase 2 multi-center, randomized, double-blind, placebo-controlled clinical study of 6R-BH4 in patients with symptomatic peripheral arterial disease (PAD). There was no statistical significance observed between the 6R-BH4 treatment and placebo groups. BioMarin will determine the future of its 6R-BH4 cardiovascular program following the results of additional investigator-sponsored studies of 6R-BH4 including proteinuria, pulmonary arterial hypertension and 6R-BH4 plus vitamin C in patients with endothelial dysfunction.

Results of First Interim Efficacy Analysis for Riquent Phase 3 ASPEN Trial: Continuation of the Trial is Futile

In February 2009, BioMarin and La Jolla Pharmaceutical (LJP) announced that in the first interim efficacy analysis (IEA) for the Riquent® Phase 3 ASPEN trial, the Independent Data Monitoring Board (DMB) determined that the continuation of the trial was futile. Following this analysis, BioMarin and La Jolla discontinued the study and BioMarin opted-out of its agreement with La Jolla to develop and commercialize Riquent in the United States, Europe and all other territories of the world, excluding the Asia Pacific region.

BioMarin & La Jolla Pharmaceutical Sign Worldwide Development and Commercialization Agreement for Riquent

In January 2009, BioMarin and La Jolla Pharmaceutical entered into an agreement to develop and commercialize Riquent®, La Jolla’s investigational drug for lupus nephritus in the United States, Europe and all other territories of the world, excluding the Asia Pacific region. If the Phase 3 trial is successful and BioMarin opts-in the parties will share equally in all losses and profits. In the United States, BioMarin and La Jolla will share Riquent commercialization rights. In Europe and other territories outside of Asia, BioMarin will have exclusive rights to commercialize the product.

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2008

Kuvan Approved By EMEA for Use in Europe

In December, the scientific committee of the European Medicines Agency (EMEA) approved Kuvan® (sapropterin dihydrochloride) Tablets as an oral treatment for hyperphenylalaninemia (HPA) in patients with phenylketonuria (PKU) or tetrahydrobiopterin (BH4) deficiency. This approval triggered a $30 million milestone payment to BioMarin from partner Merck Serono.

BioMarin Initiates Clinical Program for Morquio A Syndrome (MPS IVA)

In November, BioMarin initiated a clinical assessment program (MorCAP) for patients with MPS IVA (Morquio A Syndrome). Data collected will be used to augment current understanding of the disease by measuring endurance, respiratory function and other parameters in affected patients.

Positive Results from Study of 6R-BH4 in Sickle Cell Disease

In October, positive results from a Phase 2a multi-center, open-label, dose-escalation clinical study of 6R-BH4 in patients with sickle cell disease (SCD) indicate that oral administration of 6R-BH4 is associated with improvements in endothelial dysfunction in sickle cell disease patients. Endothelial dysfunction was measured using the EndoPAT device to assess peripheral arterial tonometry (PAT), which is commonly used in assessing the sickle cell disease patient population.

Kuvan Receives Positive Opinion from CHMP for European Approval

In September, BioMarin’s partner Merck Serono received a positive opinion for Kuvan® (sapropterin dihydrochloride) Tablets as an oral treatment for hyperphenylalaninemia (HPA) in patients with phenylketonuria (PKU) or tetrahydrobiopterin (BH4) deficiency from the Committee for Medicinal Products for Human Use (CHMP), the scientific committee of the European Medicines Agency (EMEA). The CHMP recommendation will be considered by the European Commission, which will deliver its final decision on the granting of marketing authorization within 67 days. A positive opinion will trigger a $30 million milestone payment to BioMarin.

BioMarin and Summit plc Sign Worldwide Licensing Agreement for Duchenne Muscular Dystrophy Program

In July, BioMarin and Summit Corporation partnered in an exclusive worldwide licensing agreement for Summit’s novel preclinical candidate SMT C1100 and all follow-on molecules, which are being developed to treat the fatal genetic disorder Duchenne Muscular Dystrophy (DMD).

Biopten (Sapropterin Dihydrochloride) Approved by Japanese Ministry of Health for the Treatment of PKU

In July, BioMarin partner, Asubio Pharma Co., Ltd., received marketing approval from the Japanese Ministry of Health, Labour and Welfare (MHLW) for a label extension of Biopten® (sapropterin dihydrochloride), which contains the same active ingredient as Kuvan® for the treatment of patients with phenylketonuria (PKU).

BioMarin Announces Program for ERT for Treatment of MPS IVA – Morquio A Syndrome

In June, BioMarin announced a new program for its third enzyme replacement therapy (ERT) for the treatment of mucopolysaccharidosis IVA (MPS IVA), or Morquio A Syndrome.

BioMarin Initiates Phase 1 Clinical Study of PEG-PAL in PKU

In May, the first patient initiated treatment in BioMarin’s Phase 1 clinical study of PEG-PAL (PEGylated recombinant phenylalanine ammonia lyase) for the treatment of phenylketonuria (PKU).

Naglazyme Approved by Japanese Ministry of Health

In March, AnGes MG, Inc., BioMarin’s marketing and distribution partner in Japan, received approval for its Marketing Application for Naglazyme® (galsulfase) from the Japanese Ministry of Health, Labour and Welfare (MHLW) for the treatment of patients with mucopolysaccharidosis VI ( MPS VI).

BioMarin and Genzyme Restructure Aldurazyme 50/50 Joint Venture

In January, BioMarin and Genzyme announced a restructuring of their joint venture regarding Aldurazyme® (laronidase). Genzyme will continue to globally market and sell Aldurazyme for mucopolysaccharidosis I (MPS I) and BioMarin will continue to manufacture Aldurazyme. Under the revised structure, payments are projected to result in both BioMarin and Genzyme receiving approximately the same profit as under the original joint venture structure.

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2007

BioMarin Re-Acquires Rights To Kuvan In Canada From Merck Serono

In December, BioMarin re-acquired rights to Kuvan in Canada, which will enable the company to better coordinate commercialization efforts in the North American market. Terms of the agreement specify a reduction in royalties owed to BioMarin on Merck Serono sales outside the United States and Japan

Kuvan Approved By FDA; Launched Immediately In The U.S.

In December, approximately three years after filing the IND, BioMarin received FDA approval for Kuvan® (sapropterin dihydrochloride) Tablets, the first specific drug therapy for the treatment of phenylketonuria (PKU). The product was launched in the U.S. immediately following the FDA approval. Kuvan is indicated to reduce blood phenylalanine (Phe) levels in patients with hyperphenylalaninemia (HPA) due to tetrahydrobiopterin (BH4) responsive PKU and is to be used in conjunction with a Phe-restricted diet.

BioMarin And IGAN Collaborate On Development Of Enzyme Therapy To Treat IgA Nephropathy

In December, BioMarin and IGAN Biosciences initiated a program to develop an IgA protease for treating IgA nephropathy, an orphan designated kidney disorder with few treatment alternatives. In the United States, approximately 800 patients per year develop end stage renal disease caused by IgA nephropathy. An estimated 40,000 are affected by the disorder.

BioMarin Files IND For PEG-PAL For The Treatment Of PKU

In November, BioMarin filed an investigational new drug application (IND) with the FDA for PEG-PAL (PEGylated recombinant phenylalanine ammonia lyase), formerly known as Phenylase™, for the treatment of phenylketonuria (PKU). The company expects to initiate a clinical study of PEG-PAL in PKU patients in the first quarter of 2008. Preclinical data has demonstrated that PEG-PAL administered subcutaneously once weekly to PKU mice resulted in a sustained decrease in blood phenylalanine (Phe) levels in a twelve week study and has also shown potent Phe level reductions in primates.

MAA For Sapropterin For Hyperphenylalaninemia Submitted To EMEA

In November, BioMarin partner Merck Serono, a division of Merck KGaA, Darmstadt, Germany, submitted a Marketing Authorization Application (MAA) to the European Medicines Agency (EMEA) for sapropterin dihydrochloride (known as Kuvan® in the U.S.) an oral treatment for patients suffering from significant hyperphenylalaninemia (HPA) due to phenylketonuria (PKU) or tetrahydrobiopterin (BH4) deficiency. Acceptance of the MAA filing by the EMEA will trigger a $15 million milestone payment to BioMarin. Sapropterin has received orphan medicinal product designation in the European Union.

Exclusive Rights To Kuvan Data Licensed To Asubio Pharma

In September, BioMarin has licensed exclusive rights to data and intellectual property contained in the Kuvan (sapropterin dihydrochloride) Tablets NDA to long-standing partner, Asubio Pharma Co., Ltd. (a subsidiary of Daiichi Sankyo). Asubio will use this data to supplement its current filing to the Japanese Ministry of Health, Labour and Welfare for approval of its BH4 product for the treatment of PKU in Japan. This new data greatly expands the clinical data set on treatment of PKU and is expected to accelerate the timing for the label extension of Asubio’s current BH4 product. BioMarin will receive a milestone payment for approval as well as double-digit royalties on net sales of BH4 for PKU in Japan.

BLA For Naglazyme Submitted To Japanese Ministry Of Health

In August, AnGes MG, Inc. (AnGes), BioMarin’s marketing and distribution partner in Japan submitted a Biologics License Application (BLA) for Naglazyme® (galsulfase) to the Japanese Ministry of Health, Labour and Welfare. If approved, Naglazyme will be the first drug treatment option to MPS VI patients in Japan. Naglazyme has obtained an orphan designation in Japan.

Kuvan Receives Priority Review Status From FDA

In July, priority review status is an FDA designation granted to drugs that, if approved, will provide a significant improvement in the safety or effectiveness of the treatment, diagnosis, or prevention of a serious or life-threatening disease.

BioMarin Initiates Expanded Access Program For Kuvan In The U.S.

In June, the first patient initiated treatment in BioMarin’s expanded access program for Kuvan. Under an expanded access program, the FDA allows early access to investigational drugs being developed to treat serious diseases for which there is no satisfactory alternative therapy. BioMarin will provide Kuvan at no charge to up to 500 U.S. patients throughout the duration of the program.

BioMarin Submits NDA For Kuvan For PKU

In May, the NDA filing contains data evaluating Kuvan in approximately 650 human subjects in six clinical studies and represents BioMarin’s largest and most comprehensive filing to date. The fully electronic NDA filing includes a comprehensive set of preclinical, clinical and manufacturing related data on Kuvan. If the FDA accepts the NDA and grants the request for priority review, the FDA is expected to take action on the application within six months of its submission. Kuvan has received the orphan drug designation, which allows for seven years of market exclusivity within the United States following FDA approval.

BioMarin Initiates Phase 2a Clinical Study Of 6R-BH4 In Sickle Cell Disease

In May, the first patient initiated treatment in the Phase 2a clinical study of 6R-BH4 (sapropterin dihydrochloride) for the treatment of sickle cell disease (SCD). SCD is an orphan disease with 70,000 to 100,000 patients in the U.S. It is well-diagnosed at birth, but there is only one approved drug treatment option currently available which is used by a minority of patients due to toxicity problems. The Phase 2a multi-center, open-label study will evaluate the safety of oral 6R-BH4 administered in escalating doses in patients with sickle cell disease, as well as changes in physiological and biochemical markers of endothelial function which underlie some key aspects of SCD.

Results From Phase 2 Clinical Study Of 6R-BH4 In Poorly Controlled Hypertension

In February, results demonstrated that there was no statistically significant or clinically meaningful effect of 6R-BH4 on any efficacy or safety parameter measured, relative to placebo, despite encouraging pre-clinical and clinical studies of 6R-BH4 in diseases with endothelial dysfunction.

Positive Results From Phase 3 Diet Study Of Phenoptin For PKU

In January, all pre-specified efficacy and safety endpoints of the double-blind, placebo-controlled Phase 3 diet study of Phenoptin™ (sapropterin dihydrochloride) were met. Treatment resulted in a significant increase in patients’ phenylalanine tolerance as well as a reduction in their blood phenylalanine levels. In addition, the data showed that Phenoptin was well tolerated in younger PKU patients under dietary control.

Phase 2 Clinical Study Of 6R-BH4 In Peripheral Arterial Disease Initiated

In January, the first patient initiated treatment in the Phase 2 clinical study of 6R-BH4 for the treatment of symptomatic peripheral arterial disease. The company expects to announce data from this study in the first half of fiscal year 2008. Peripheral arterial disease results from endothelial dysfunction and affects approximately eight million Americans, many of whom also suffer from intermittent claudication. The Phase 2, multicenter, multinational, randomized, double-blind, placebo-controlled study is designed to compare oral 6R-BH4 to placebo in subjects with intermittent claudication (IC) caused by peripheral arterial disease.

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2006

Positive Results From Phase 3 Extension Study Of Phenoptin For PKU

In December, data has confirmed the long-term safety, tolerability, and efficacy of Phenoptin to control blood Phe levels across a range of doses in PKU patients. The company is on track to file the NDA in the second quarter of 2007. A once daily dose regimen of Phenoptin was sufficient to maintain the reduction of blood Phe levels throughout a 24-hour period. The incidence and type of adverse events were comparable to that of the placebo group during the double-blind study and nearly all were mild or moderate in severity.

Aldurazyme Receives Marketing Approval In Japan

In October, Japan’s Ministry of Health, Labor, and Welfare (MHLW) has granted marketing authorization for Aldurazyme® (laronidase), the first specific treatment for MPS I approved in Japan. Aldurazyme has been designated as an orphan drug in Japan.

Orapred ODT Launched By Alliant Pharmaceuticals

In August, Orapred ODT™ (prednisolone sodium phosphate orally disintegrating tablets), the first FDA-approved orally disintegrating tablet form of prednisolone, is now available in the United States. The Orapred product line, which includes Orapred ODT and Orapred® (prednisolone sodium phosphate oral solution) is marketed by Alliant Pharmaceuticals, Inc. Under the terms of the marketing agreement, BioMarin will receive milestone payments and royalties on Orapred products sales. BioMarin will retain commercial rights to the Orapred product line outside of North America.

Phase 2 Clinical Study Of 6R-BH4 In Poorly Controlled Hypertension Initiated

In July, the first patient initiated treatment in the Phase 2 clinical study of 6R-BH4 for the treatment of poorly controlled hypertension. The company hopes to announce data from this study in early 2007 that will confirm results seen earlier in pilot clinical studies that demonstrated that oral administration of 6R-BH4 can reduce blood pressure in individuals who remain hypertensive despite treatment with other medications.

BioMarin And Alliant Pharmaceuticals Establish North American Licensing Agreement For Orapred

In March, the licensing and acquisition agreement provides exclusive North American rights to Alliant for the Orapred® (prednisolone sodium phosphate oral solution) product line, including Orapred ODT™ (prednisolone sodium phosphate orally disintegrating tablets). BioMarin will receive payments and royalties based on the product’s approval, launch and level of sales. BioMarin will retain commercial rights outside of North America

Positive Results From Phase 3 Clinical Study Of Phenoptin For PKU

In March, positive results of a Phase 3, double-blind, placebo-controlled clinical study of Phenoptin™ (sapropterin dihydrochloride) confirmed that all pre-specified primary and secondary endpoints were met. Data demonstrates a statistically significant reduction at six weeks in blood phenylalanine (Phe) levels (p<0.0001) in patients receiving Phenoptin, compared with those receiving placebo.

Naglazyme Receives European Union Approval

In January, the European Commission has granted marketing authorization for Naglazyme® (galsulfase), the first treatment for MPS VI approved in the European Union. Naglazyme has been granted orphan drug status in the EU, which confers 10 years of market exclusivity. BioMarin will launch the product on a country-by-country basis.

Phenoptin For PKU Receives FDA Fast Track Designation

In January, the U.S. Food and Drug Administration (FDA) granted Fast Track designation for Phenoptin™ (sapropterin dihydrochloride) for PKU, currently in Phase 3 clinical development.

The Fast Track program is designed to expedite the development and review process of new drugs that are intended to treat serious or life-threatening conditions and that demonstrate the potential to address unmet medical needs.

BioMarin Establishes Commercial Operations
In Europe

In January, in anticipation of the pending European marketing approval for Naglazyme™, commercial operations have been established in Europe. BioMarin Europe Ltd., headquartered in London, with branch offices located in Spain, Switzerland and Italy, will be responsible for overseeing the sales and distribution of Naglazyme to the 25 member states of the European Union, Iceland and Norway.

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2005

BioMarin Files New Drug Application For
Orapred ODT

In August, BioMarin submitted a New Drug Application to the FDA for Orapred ODT™ (prednisolone sodium phosphate orally disintegrating tablets), a new formulation of Orapred® (prednisolone sodium phosphate oral solution). Prednisolone is commonly used to reduce inflammation seen in numerous medical conditions including asthma, arthritis, and cancer.

Orapred ODT may have the potential to provide individuals of all ages with a new formulation of prednisolone that is convenient and easy to administer.

BioMarin Launches Naglazyme In The United
States


In June, Naglazyme® is the first drug therapy independently developed and commercialized by BioMarin. A team of U.S. based medical science liaisons will be responsible for providing support to infusion centers and physicians who administer Naglazyme.

BioMarin Receives FDA Approval For Naglazyme

In May, Naglazyme®, represents BioMarin’s first independently developed and commercialized drug and the first FDA-approved treatment for MPS VI. The drug was granted the orphan drug designation in the United States, which confers seven years of market exclusivity.


BioMarin Forms Strategic Alliance With Serono for the Development and Commercialization of Phenoptin and Phenylase

In May, BioMarin and Serono finalize an egreement to help fund late-stage development of BioMarin’s PKU and 6R-BH4 programs. The companies will equally share Phase 3 development costs of Phenoptin and Phenylase for PKU and 6R-BH4 for the treatment of cardiovascular indications. Additionally, Serono will provide BioMarin up to $232 million in milestone payments in exchange for ex-U.S. commercialization rights (excluding Japan).

BioMarin Acquires Rights to 6R-BH4 for the Treatment of Cardiovascular Indications

In May, BioMarin announced a partnership with Daiichi Suntory Pharma Co., Ltd. that gave the company exclusive worldwide rights (excluding Japan) for the use of 6R-BH4 to treat cardiovascular indications. This was the second agreement reached with Daiichi; the first, which was reached in November 2004, pertained to intellectual property, preclinical and clinical data on 6R-BH4 for genetic disorders including PKU, and to manufacturing and supply of 6R-BH4.

BioMarin Initiates Phase 3 Clinical Study of Phenoptin for PKU

In April, BioMarin initiated a Phase 3 clinical study designed to evaluate the safety and efficacy of Phenoptin for the treatment of PKU. Phenoptin has been designated an orphan drug in the United States and Europe and assigned Fast Track status in the United States.

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2004

BioMarin Files Marketing Applications for Naglazyme in the United States and Europe

In June, BioMarin announced positive results of the Phase 3 clinical trial of Naglayzme (galsulfase) for MPS VI, keeping the company on track to file license applications in both the United States and European Union by the close of the year.

BioMarin Advances Phenoptin for PKU into Phase 2 Development

In February, BioMarin advanced Phenoptin™ (sapropterin dihydrochloride), an investigational small-molecule oral therapeutic for the treatment of phenylketonuria (PKU), from IND filing into Phase 2 development by the close of the year.

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2003

Aldurazyme for MPS I Approved and Launched in the United States and Europe

In April and June, respectively, the FDA and European Commission (EC) granted marketing authorization for Aldurazyme–the first approved enzyme replacement therapy for the treatment of MPS I. Aldurazyme received FDA approval in just over five and a half years after the investigational new drug application (IND) was filed.

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2000

BioMarin Initiates Clinical Trial of Naglazyme for MPS VI

At the onset of clinical development, Naglazyme® (galsulfase) for MPS VI had been granted orphan drug and fast track designations by the U.S. Food and Drug Administration (FDA). The company manufactured the investigational enzyme at its cGMP manufacturing facility located near the large-scale facility where it is being manufactured today.

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1999

BioMarin Becomes a Publicly Traded Company (Nasdaq/SWX:BMRN)

In July, BioMarin completed an initial public offering, raising $67.3 million. Since a large number of the early investors were based in Europe, the company was listed on the Swiss SWX Exchange in addition to the Nasdaq National Market.

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1998

BioMarin/Genzyme LLC Formed to Support the Development and Commercialization of Aldurazyme

In September, BioMarin and Genzyme Corporation established BioMarin/Genzyme LLC, a 50/50 joint venture for the worldwide development and commercialization of Aldurazyme for MPS I. Pursuant to the agreement, BioMarin is responsible for manufacturing the product and Genzyme is responsible for its commercialization. All expenses and profits and are shared equally between the companies.

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1997

BioMarin Initiates Clinical Trial of Aldurazyme for MPS I

In December, BioMarin initiated the first clinical trial of Aldurazyme® (laronidase) for the treatment of mucopolysaccharidosis I (MPS I). Dr. Emil Kakkis, then a fellow at Harbor-UCLA Medical Center, was the study’s principal investigator and Dr. Elizabeth Neufeld, Professor and Chair of the Biological Chemistry Department at UCLA, was the advisor. Together, Dr. Neufeld and Dr. Kakkis discovered how to produce a recombinant form of alpha-L-iduronidase that was later evaluated in the clinic.

BioMarin Pharmaceutical Inc. Founded

In March, with a $1.5 million investment from Glyko Biomedical Ltd., BioMarin was open for business. The company’s mission was to leverage its proprietary enzyme technology to develop therapies for the treatment of numerous diseases and conditions including genetic diseases, and burn and wound care. By the close of the year, the company had raised an additional $11.3 million from private investors.

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Image: Jean-Jacques Bienaimé - BioMarin CEO
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